title: A fludarabine-based protocol for bone marrow transplantation in Fanconi's anemia.
abstract: Allogeneic bone marrow transplantation (BMT) is an effective therapy for Fanconi's anemia (FA). However, mortality and transplant-related complications are usually high due to increased sensitivity to the alkylating agents and radiation commonly used for pre-transplant conditioning. Fludarabine monophosphate is a purine analogue that has been proven effective as a conditioning agent for chronic lymphocytic leukemia patients. We report a child with FA in leukemic transformation with thrombocytopenia and 20% myeloblasts who underwent successful BMT following conditioning with fludarabine/ATG/cyclophosphamide. The regimen was well tolerated, no transplant-related complications were observed, and engraftment was rapid. The child is currently 10 months post-BMT, in excellent clinical condition with a normal blood count, 100% chimerism and no sign of graft-versus-host disease (GVHD). We suggest that this fludarabine-based regimen may be effective in the conditioning of standard, as well as transforming, FA patients for BMT.
authors.affiliation: Department of Bone Marrow Transplantation, Hadassah University Hospital, Jerusalem, Israel.
authors.forename: J
authors.initials: J
authors.lastname: Kapelushnik
authors.forename: R
authors.initials: R
authors.lastname: Or
authors.forename: S
authors.initials: S
authors.lastname: Slavin
authors.forename: A
authors.initials: A
authors.lastname: Nagler
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D002648:Child; D003520:Cyclophosphamide / Q000008:administration & dosage / Q000627:therapeutic use; D005199:Fanconi Anemia / Q000188:drug therapy / Q000628:therapy*; D005260:Female; D006801:Humans; D007951:Leukemia, Myeloid / Q000209:etiology / Q000628:therapy*; D008297:Male; D011289:Preleukemia / Q000209:etiology / Q000628:therapy*; D012074:Remission Induction; D012307:Risk Factors; D019172:Transplantation Conditioning; D014740:Vidarabine / Q000031:analogs & derivatives* / Q000627:therapeutic use
chemical_list: D000970:Antineoplastic Agents; D003520:Cyclophosphamide; D014740:Vidarabine; C024352:fludarabine